NCT05040178 An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
| NCT ID | NCT05040178 |
| Status | Recruiting |
| Phase | — |
| Sponsor | RECORDATI GROUP |
| Condition | Hyperammonemia |
| Study Type | OBSERVATIONAL |
| Enrollment | 20 participants |
| Start Date | 2022-06-30 |
| Primary Completion | 2032-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 20 participants in total. It began in 2022-06-30 with a primary completion date of 2032-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Eligibility Criteria
Inclusion Criteria: 1. Provision of signed and dated informed consent/assent form 2. Prescribed and treated with Carbaglu® 3. Have an established diagnosis of PA or MMA defined as follows: * Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR * Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness). AND/OR * Confirmation by molecular genetic testing Exclusion Criteria: * None
Contact & Investigator
William Ludlum, MD
STUDY DIRECTOR
Recordati Rare Diseases Inc
Frequently Asked Questions
Who can join the NCT05040178 clinical trial?
This trial is open to participants of all sexes, studying Hyperammonemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05040178 currently recruiting?
Yes, NCT05040178 is actively recruiting participants. Contact the research team at cesario.a@recordati.com for enrollment information.
Where is the NCT05040178 trial being conducted?
This trial is being conducted at Washington D.C., United States, Tampa, United States, Chicago, United States, Indianapolis, United States and 1 additional location.
Who is sponsoring the NCT05040178 clinical trial?
NCT05040178 is sponsored by RECORDATI GROUP. The principal investigator is William Ludlum, MD at Recordati Rare Diseases Inc. The trial plans to enroll 20 participants.